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Rodríguez-Lebrón et al., 2013 - Google Patents

  • ️Tue Jan 01 2013
Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice

Rodríguez-Lebrón et al., 2013

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Document ID
10057236992865500594
Author
doCarmo Costa M
Luna-Cancalon K
Peron T
Fischer S
Boudreau R
Davidson B
Paulson H
Publication year
2013
Publication venue
Molecular Therapy

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Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disease caused by a polyglutamine expansion in the deubiquitinating enzyme, Ataxin-3. Currently, there are no effective treatments for this fatal disorder but studies support the hypothesis that reducing …

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